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ClinicalTrials.gov public records Last synced Nov 15, 2026, 4:57 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 24 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Gaucher Disease, Type 3 Clear all

Conditions: Gaucher Disease
Interventions: Eliglustat tartrate; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 170 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 10
States / cities: San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more

Conditions: Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions: Velaglucerase alfa; Drug
Lead sponsor: Baylor Research Institute; Other
Eligibility: 4 Years to 14 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Gaucher Disease, Type 1
Interventions: Eliglustat tartrate, Placebo; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 6
States / cities: San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more

Conditions: Gaucher Disease
Interventions: OGT 918; Drug
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: 4 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2007
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gaucher Disease, Type 3
Interventions: No intervention; Other
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years to 80 Years
Enrollment: 5 participants
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Gaucher Disease
Interventions: Taliglucerase alfa; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 2 Years and older
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 3
States / cities: Coral Springs, Florida • Decatur, Georgia • New York, New York

Conditions: Gaucher's Disease
Interventions: Lysodase; Drug
Lead sponsor: National Institute of Mental Health (NIMH); NIH
Eligibility: Not listed
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1993 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gaucher Disease, Type 1
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 1
States / cities: New York, New York

Conditions: Melanoma
Interventions: KLH conjugates with GD2L and GD3L; Biological
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 21 Years and older
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: New York, New York

Conditions: Type 2 Gaucher Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Up to 5 Years · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Neuroblastoma
Interventions: adjuvant OPT-821 in a vaccine containing two antigens (GD2L and GD3L) covalently linked to KLH, oral β-glucan; Biological
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: Up to 21 Years
Enrollment: 374 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Gaucher's Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1991 – 2008
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gaucher Disease Type 1
Interventions: FLT201; Genetic
Lead sponsor: Spur Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 15
States / cities: Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions: There is no intervention; Other
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 64 Years
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2050
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Melanoma
Interventions: PF-06688992; Drug
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 1
States / cities: New York, New York

Conditions: Gaucher's Disease Type III
Interventions: Venglustat, imiglucerase; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 4
States / cities: New Haven, Connecticut • Iowa City, Iowa • Dallas, Texas + 1 more

Conditions: Gaucher Disease, Type 1
Interventions: Eliglustat tartrate, Imiglucerase; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2015
U.S. locations: 17
States / cities: Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more

Conditions: Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions: venglustat (GZ402671), imiglucerase; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 3
States / cities: New Haven, Connecticut • Dallas, Texas • Fairfax, Virginia

Conditions: Sarcoma
Interventions: Trivalent ganglioside vaccine, OPT-821; Biological
Lead sponsor: MabVax Therapeutics, Inc.; Industry
Eligibility: 16 Years and older
Enrollment: 136 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2017
U.S. locations: 13
States / cities: Los Angeles, California • Denver, Colorado • Atlanta, Georgia + 10 more

Conditions: Stage IV Melanoma
Interventions: KW-2871; Drug
Lead sponsor: Kyowa Kirin, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 2
States / cities: Birmingham, Alabama • Tampa, Florida

Conditions: Gaucher Disease
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Metastatic Melanoma, Cutaneous Melanoma
Interventions: HDI, KW2871; Drug
Lead sponsor: Ludwig Institute for Cancer Research; Other
Eligibility: 18 Years and older
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2014
U.S. locations: 2
States / cities: Chicago, Illinois • Pittsburgh, Pennsylvania

Conditions: Gaucher Disease
Interventions: Blood sample; Procedure
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: Not listed
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 7
States / cities: San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 4 more

Search by objective public record fields.

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Determine Toxicity and Antibody Responses With a KLH Conjugated Bivalent Vaccine Containing GD2 Lactone, GD3 Lactone With Immunological Adjuvant QS-DG or OPT-821 in Patients With Disease Free AJCC Stage III or IV Cutaneous Melanoma

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

Bivalent Vaccine With Escalating Doses of the Immunological Adjuvant OPT-821, in Combination With Oral β-glucan for High-Risk Neuroblastoma

Effects of Enzyme Replacement in Gaucher's Disease

A Gaucher Disease Gene Therapy Trial With FLT201

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Pfizer PF-06688992 in Patients With Stage III or Stage IV Melanoma

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Trivalent Ganglioside Vaccine With Immunological Adjuvant or Immunological Adjuvant Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free

A Study of a Monoclonal Antibody, KW-2871, in Patients With Advanced Melanoma

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Phase II Study of KW2871 Combined With High Dose Interferon-α2b in Patients With Metastatic Melanoma

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease